Slide 1: Courageous Innovation: Gene & Cell Therapies and Vaccines
Courageous Innovation Dedicated to Bringing Game - Changing Gene & Cell Therapies and Vaccines to Market and Working Even Harder to Provide Access to Patients Globally OCGN August 2024
Slide 2: Forward Looking Statements
Forward Looking Statements 2 This presentation contains forward - looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “co
Slide 3: Leveraging Our First-in-Class Platforms to Address Serious Unmet Medical Needs
Through Courageous Innovation, We are Leveraging Our First - in - Class Platforms to Address Serious Unmet Medical Needs 3 M odifier G ene T herapy Platform First - in - Class Inhalation Vaccines Platform First - in - Class Regenerative Cell Therapy Platform First - in - Class • Therapeutic Focus: I
Slide 4: Pipeline Overview
4 Pipeline Overview Asset/Program Indication Current Status Gene therapies OCU400 * AAV - hNR2E3 Retinitis pigmentosa (RP) — RHO Mutations • Actively dosing subjects in the OCU400 Phase 3 liMeliGhT trial for the treatment of RP • FDA granted RMAT** designation for RP indication associated with NR2E3
Slide 5: Modifier Gene Therapy Platform
5 Modifier Gene Therapy Platform Breakthrough technology designed to address many rare diseases as well as complex diseases that affect millions
Slide 6: Traditional Gene Therapy is Highly Inefficient for Retinal Diseases
• 13,120 (65%) of all human proteins expressed in retina • 785 genes are highly specialized in the retina • Nearly 300 mutations have been identified • 100+ associated with RP • 1 gene therapy for a single mutation is approved (2017) Traditional single therapy is not efficient for disease such as RP
Slide 7: Photoreceptor Dependent on Cellular Homeostasis Regulated by Transcription Factors such as NR2E3
The Photoreceptor is Dependent on Cellular Homeostasis Regulated by Key Transcription Factors (TFs) such as NR2E3 (OCU400) 1 Jones BW, Marc RE, Pfeiffer RL. Retinal Degeneration, Remodeling and Plasticity. 2016 Oct 28. In: Kolb H, Fernandez E, Nelson R, editors. Webvision : The Organization of the R
Slide 8: What is Modifier Gene Therapy?
What is Modifier Gene Therapy? • The insertion of a nuclear hormone receptor gene (transcription factor) to regulate the expression of multiple genes required for cell survival & maintenance Animal Proof of Concept – Gene Agnostic Mechanism • OCU400 ( NR2E3 ) demonstrated restoration of gene express
Slide 9: Regulatory Achievements
Regulatory Achievements • Received RMAT Designation from the FDA • FDA/EMA granted expanded orphan drug designations for RP • EMA provided acceptability of the U.S. - based trial for submission of MAA • EAP for the treatment of adult patients, aged 18 and older, with RP • A Despite its prevalence, R
Slide 10: Human Proof of Concept: Phase 1/2 Safety and Efficacy Summary
• OCU400 is observed to be generally well - tolerated and effective in subjects across different mutations and dose levels — 18 patients • Efficacy measurements suggest stabilization or improvement in the treated eye on validated mobility test course (score vs baseline) regardless of mutation • 78%
Slide 11: Mobility Test (LDNA) is Primary Endpoint for Phase 3
11 Mobility Test (LDNA*) is Primary Endpoint for Phase 3 11 • Primary endpoint (Efficacy): Mobility test has been used for an approved product • Alignment with FDA to demonstrate clinical efficacy (Responder ≥ 2 Lux level improvement) - Validated for RP patients • Phase 1/2 results: More than 60% of
Slide 12: OCU400: Expected Pathway to Clinical Development & Potential Approval
RP LCA Adult & Pediatric • Both FDA & EMA granted broad orphan drug designation for RP & LCA • Received RMAT designation from FDA RHO NR2E3 CEP290 Indication Expanded Access Program for Broad RP approved by FDA Treatment of RP OCU400: Expected Pathway to Clinical Development & Potential Approval 12
Slide 13: OCU410 (RORA): A Single-Injection Approach to Addressing Unmet Needs in dAMD
MOA: Mechanism of action | nAMD : Neovascular age - related macular degeneration 13 • Advancement from recently approved therapies for GA: Potential to address limitations of recently approved therapies for GA focused only on the complement system, including: • Patient Compliance • Frequent intravit
Slide 14: OCU410 (RORA): A Potential Modifier Therapeutic for Dry-AMD and STGD
50 60 70 80 90 1e6 1E5 1E4 1E3 1E2 Cells Normalized Cell Survival RORA NR2E3 NR1D1 Anti - oxidative: Improves ARPE19 cell survival Anti - complement: Increases anti - complement (Cd59) protein Anti - inflammatory: Suppresses inflammation in HMC3 cells Anti - drusen activity: Improves retinal functio
Slide 15: OCU410ST: Received ODD for ABCA4-Associated Retinopathies
15 OCU410ST: Received ODD for ABCA4 - Associated Retinopathies: Stargardt, Retinitis Pigmentosa 19 & Cone - rod Dystrophy 3 ABCA4 - associated retinopathies — Genetic Rare Disease • ABCA4 gene produces an ATP - binding cassette (ABC) superfamily transmembrane protein involved in clearance of all - t
Slide 16: NeoCart: Autologous chondrocyte-derived neocartilage
16 NeoCart ® Autologous chondrocyte - derived neocartilage
Slide 17: NeoCart is a regenerative cell therapy
17 *Journal of Bone & Joint Surgery: June 1, 2011 - Volume 93 - Issue 11 - p 994 - 1000 **https://www.biospace.com/article/cell - therapy - market - size - cagr - trends - forecast - report - 2022 - 2030/ NeoCart is a regenerative cell therapy • Received RMAT designation • Combines bioengineering an
Slide 18: OCU500 Series
18 OCU500 Series OCU500: COVID - 19 Mucosal Vaccine OCU510: Flu OCU520: COVID - 19/Flu
Slide 19: OCU500 Series: Next-Generation Vaccine Technology
19 OCU500 Series: Next - Generation Vaccine Technology Inhaled mucosal vaccine platform based on ChAd vector OCU500 COVID - 19 vaccine OCU510 Seasonal quadrivalent flu vaccine OCU520 Combination quadrivalent flu and COVID - 19 vaccine Inhalation technology as a differentiator • Multiple preclinical
Slide 20: 2024 Near-Term Targeted Milestones
20 2024 Near - Term Targeted Milestones • OCU400 Phase 3 dosing and recruitment updates – 2026 BLA/MAA approval targets on track • Preliminary safety/efficacy updates – OCU410 Phase 1/2 clinical trial (GA) • Preliminary safety/efficacy updates – OCU410ST Phase 1/2 clinical trial (Stargardt Disease)
Slide 21: Ocugen Vision
21 Ocugen Ρ Vision Fully integrated, patient - centric b iotech company focused on vaccines in support of public health and gene and cell therapies targeting unmet medical needs through Courageous Innovation